Aplastic Anemia: A Rare Disease With a Better Prognosis

SUNDAY, Feb. 4 (HealthDay News) -- Aplastic anemia, a disease of the bone marrow, is a rare disorder in the United States. Only three of every 1 million Americans will be diagnosed with the condition this year, the National Marrow Donor Program reports.

Despite that rarity, this once-fatal disease has become far more treatable as physicians have honed in on practices that can prolong life and ease suffering.

And the effects of that research extend far beyond sufferers of aplastic anemia or other related bone marrow diseases. Insights gained from these diseases are also helping scientists learn about more prevalent health problems, such as heart disease or leukemia, researchers say.

Aplastic anemia occurs when bone marrow stops producing enough blood cells, said Katherine Baer, a patient information specialist for the Aplastic Anemia and MDS International Foundation Inc. Only about 1,000 new cases appear each year in the United States.

A related blood disorder, myelodysplastic syndromes, or MDS, occurs when the bone marrow begins producing poorly functioning or immature blood cells. About 20,000 to 30,000 new cases occur each year.

Doctors still aren't certain exactly what causes the diseases' onset, Baer said.

"They do think it can be caused; there are some toxins that may cause it, like benzene," Baer said. "But at least half the cases are of unknown cause." She added that radiation treatments for other diseases are another suspected cause.

The effects of aplastic anemia and MDS vary, depending on the type of blood cells lacking in the body, Baer said.

Red blood cells carry oxygen, and a shortage of those will cause fatigue and shortness of breath. White blood cells fight infection, so when the body lacks those cells, it is more likely to catch infectious diseases. Platelets cause clotting, and without those, people experience nosebleeds, bleeding gums and extended bleeding from cuts.

These diseases used to be killers, fatal within a year, said Dr. Richard Stone, clinical director of the Adult Leukemia Program at Harvard University's Dana-Farber Cancer Institute and an associate professor at Harvard Medical School.

"Now, people can be expected to live a long time in many cases," Stone said. "It's devastating if untreated but quite approachable if treated."

Baer and Stone said that while no breakthrough treatments have been developed, the available therapies are at the point where people can live with the disorders.